Scientists hunt for clues to slow rare brain disease
NCT ID NCT00344331
First seen Jun 24, 2026 · Last updated Jun 24, 2026
Summary
This study follows 900 people with Niemann-Pick Type C, a rare genetic disorder that damages the nervous system. Researchers will run tests every six months to find biological markers that show how the disease progresses. No experimental drug is given, but the results may help design future treatment studies.
Disclaimer
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This is a summary of
the original study
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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National Institutes of Health Clinical Center
RECRUITINGBethesda, Maryland, 20892, United States
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
What this could lead to
If successful, this study could identify reliable markers to measure disease progression, paving the way for future treatment trials.
What could go wrong
This is an observational study, not a treatment trial. It may not find useful markers, and results may not apply to all patients.
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.